KCNQ1 suppression-replacement gene therapy in transgenic rabbits with type 1 long QT syndrome.
Sahej BainsLucilla GiammarinoSaranda NimaniNicolo AlerniDavid J TesterC S John KimNicolas ChristoforouJulien LouradourAndrás HorváthOlgica BeslacMiriam BarbieriLluis MatasThomas S HofRuben LopezStefanie Perez-FelizChiara ParodiLuisana G Garcia CasaltaJacqulyn JurgensenMichael A BarryMariana BegoLisa KeyesJane OwensJason PinkstaffGideon KorenManfred ZehenderMichael BrunnerDaniela CasoniFabien PrazAndreas HaeberlinGabriel BrooksMichael J AckermanKatja E OdeningPublished in: European heart journal (2024)
This study provides the first animal-model, proof-of-concept gene therapy for correction of LQT1. In LQT1 rabbits, treatment with KCNQ1-SupRep gene therapy normalized the clinical QTi and cellular APD90 to near WT levels both at baseline and after isoproterenol. If similar QT/APD correction can be achieved with intravenous administration of KCNQ1-SupRep gene therapy in LQT1 rabbits, these encouraging data should compel continued development of this gene therapy for patients with LQT1.